Ristoglogene Autogetemcel Medicine 04 June 2026 Extended indication Ristoglogene autogetemcel monotherapy for treatment of severe Sickle cell disease in adolescents and adults up to 35 years of age following myeloablative conditioning with busulfan who have severe vaso-occlusive crises Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Ristoglogene Autogetemcel Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication Ristoglogene autogetemcel monotherapy for treatment of severe Sickle cell disease in adolescents and adults up to 35 years of age following myeloablative conditioning with busulfan who have severe vaso-occlusive crises Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Portfolio holder Mechanism of action Autologous modified cell therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Fabrikant: Beam Therapeutics Registration Registration route Type of trajectory Particularity ATMP Submission date January 2027 Expected Registration February 2028 Orphan drug Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Transfusies met hydroxyurea/hydroxycarbamide en rode bloedcellen (aanvullen/wisselen) Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT05456880 (fase 1/2, BEACON) Additional remarks Toediening via intraveneuze infusie na myeloablatieve conditionering met busulfan bij patiënten met ernstige vaso-occlusieve crises. Expected patient volume per year Patient volume < 600 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References (1) https://www.sanquin.nl/donors/alles-over-bloed/bloedziektes/sikkelcelziekte. (2) Arlet J, Herquelot E, Lamarsalle L et al. (2024) https://doi.org/10.1016/j.lanepe.2024.100901 Additional remarks In Nederland hebben ongeveer 2.000 mensen de diagnose sikkelcelziekte (1). Ongeveer 30% van de patiënten heeft meer dan een hospitalized vaso-occlusive crisis (HVOC) per jaar (2). Expected cost per patient per year Cost References Additional remarks Er is nog niets bekend over de mogelijke prijs. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks